Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown
Wittrup, Anders, Ai, Angela, Liu, Xing, Hamar, Peter, Trifonova, Radiana, Charisse, Klaus, Manoharan, Muthiah, Kirchhausen, Tomas, Lieberman, Judy
Published in Nature biotechnology (01.08.2015)
Published in Nature biotechnology (01.08.2015)
Get full text
Journal Article
Multivalent N‑Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
Nair, Jayaprakash K., Willoughby, Jennifer L. S., Chan, Amy, Charisse, Klaus, Alam, Md. Rowshon, Wang, Qianfan, Hoekstra, Menno, Kandasamy, Pachamuthu, Kel’in, Alexander V., Milstein, Stuart, Taneja, Nate, O’Shea, Jonathan, Shaikh, Sarfraz, Zhang, Ligang, van der Sluis, Ronald J., Jung, Michael E., Akinc, Akin, Hutabarat, Renta, Kuchimanchi, Satya, Fitzgerald, Kevin, Zimmermann, Tracy, van Berkel, Theo J. C., Maier, Martin A., Rajeev, Kallanthottathil G., Manoharan, Muthiah
Published in Journal of the American Chemical Society (10.12.2014)
Published in Journal of the American Chemical Society (10.12.2014)
Get full text
Journal Article
Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates
Nair, Jayaprakash K, Attarwala, Husain, Sehgal, Alfica, Wang, Qianfan, Aluri, Krishna, Zhang, Xuemei, Gao, Minggeng, Liu, Ju, Indrakanti, Ramesh, Schofield, Sally, Kretschmer, Philip, Brown, Christopher R, Gupta, Swati, Willoughby, Jennifer L S, Boshar, Julie A, Jadhav, Vasant, Charisse, Klaus, Zimmermann, Tracy, Fitzgerald, Kevin, Manoharan, Muthiah, Rajeev, Kallanthottathil G, Akinc, Akin, Hutabarat, Renta, Maier, Martin A
Published in Nucleic acids research (02.11.2017)
Published in Nucleic acids research (02.11.2017)
Get full text
Journal Article
An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia
Sehgal, Alfica, Barros, Scott, Ivanciu, Lacramioara, Cooley, Brian, Qin, June, Racie, Tim, Hettinger, Julia, Carioto, Mary, Jiang, Yongfeng, Brodsky, Josh, Prabhala, Harsha, Zhang, Xuemei, Attarwala, Husain, Hutabarat, Renta, Foster, Don, Milstein, Stuart, Charisse, Klaus, Kuchimanchi, Satya, Maier, Martin A, Nechev, Lubo, Kandasamy, Pachamuthu, Kel'in, Alexander V, Nair, Jayaprakash K, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Meyers, Rachel, Sorensen, Benny, Simon, Amy R, Dargaud, Yesim, Negrier, Claude, Camire, Rodney M, Akinc, Akin
Published in Nature medicine (01.05.2015)
Published in Nature medicine (01.05.2015)
Get full text
Journal Article
From bench to bedside: Improving the clinical safety of GalNAc–siRNA conjugates using seed-pairing destabilization
Schlegel, Mark K, Janas, Maja M, Jiang, Yongfeng, Barry, Joseph D, Davis, Wendell, Agarwal, Saket, Berman, Daniel, Brown, Christopher R, Castoreno, Adam, LeBlanc, Sarah, Liebow, Abigail, Mayo, Tara, Milstein, Stuart, Nguyen, Tuyen, Shulga-Morskaya, Svetlana, Hyde, Sarah, Schofield, Sally, Szeto, John, Woods, Lauren Blair, Yilmaz, Vedat O, Manoharan, Muthiah, Egli, Martin, Charissé, Klaus, Sepp-Lorenzino, Laura, Haslett, Patrick, Fitzgerald, Kevin, Jadhav, Vasant, Maier, Martin A
Published in Nucleic acids research (08.07.2022)
Published in Nucleic acids research (08.07.2022)
Get full text
Journal Article
Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression
Willoughby, Jennifer L.S., Chan, Amy, Sehgal, Alfica, Butler, James S., Nair, Jayaprakash K., Racie, Tim, Shulga-Morskaya, Svetlana, Nguyen, Tuyen, Qian, Kun, Yucius, Kristina, Charisse, Klaus, van Berkel, Theo J.C., Manoharan, Muthiah, Rajeev, Kallanthottathil G., Maier, Martin A., Jadhav, Vasant, Zimmermann, Tracy S.
Published in Molecular therapy (03.01.2018)
Published in Molecular therapy (03.01.2018)
Get full text
Journal Article
2′-Fluoro RNA Shows Increased Watson-Crick H-Bonding Strength and Stacking Relative to RNA: Evidence from NMR and Thermodynamic Data
Patra, Amritraj, Paolillo, Michael, Charisse, Klaus, Manoharan, Muthiah, Rozners, Eriks, Egli, Martin
Published in Angewandte Chemie International Edition (19.11.2012)
Published in Angewandte Chemie International Edition (19.11.2012)
Get full text
Journal Article
Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-nucleosidic Trivalent N-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo
Rajeev, Kallanthottathil G., Nair, Jayaprakash K., Jayaraman, Muthusamy, Charisse, Klaus, Taneja, Nate, O'Shea, Jonathan, Willoughby, Jennifer L. S., Yucius, Kristina, Nguyen, Tuyen, Shulga-Morskaya, Svetlana, Milstein, Stuart, Liebow, Abigail, Querbes, William, Borodovsky, Anna, Fitzgerald, Kevin, Maier, Martin A., Manoharan, Muthiah
Published in Chembiochem : a European journal of chemical biology (13.04.2015)
Published in Chembiochem : a European journal of chemical biology (13.04.2015)
Get full text
Journal Article
siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes
Matsuda, Shigeo, Keiser, Kristofer, Nair, Jayaprakash K, Charisse, Klaus, Manoharan, Rajar M, Kretschmer, Philip, Peng, Chang G, V. Kel’in, Alexander, Kandasamy, Pachamuthu, Willoughby, Jennifer L.S, Liebow, Abigail, Querbes, William, Yucius, Kristina, Nguyen, Tuyen, Milstein, Stuart, Maier, Martin A, Rajeev, Kallanthottathil G, Manoharan, Muthiah
Published in ACS chemical biology (15.05.2015)
Published in ACS chemical biology (15.05.2015)
Get full text
Journal Article
FGF Regulates TGF-β Signaling and Endothelial-to-Mesenchymal Transition via Control of let-7 miRNA Expression
Chen, Pei-Yu, Qin, Lingfeng, Barnes, Carmen, Charisse, Klaus, Yi, Tai, Zhang, Xinbo, Ali, Rahmat, Medina, Pedro P., Yu, Jun, Slack, Frank J., Anderson, Daniel G., Kotelianski, Victor, Wang, Fen, Tellides, George, Simons, Michael
Published in Cell reports (Cambridge) (27.12.2012)
Published in Cell reports (Cambridge) (27.12.2012)
Get full text
Journal Article
Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates
Frank-Kamenetsky, Maria, Grefhorst, Aldo, Anderson, Norma N, Racie, Timothy S, Bramlage, Birgit, Akinc, Akin, Butler, David, Charisse, Klaus, Dorkin, Robert, Fan, Yupeng, Gamba-Vitalo, Christina, Hadwiger, Philipp, Jayaraman, Muthusamy, John, Matthias, Jayaprakash, K. Narayanannair, Maier, Martin, Nechev, Lubomir, Rajeev, Kallanthottathil G, Read, Timothy, Röhl, Ingo, Soutschek, Jürgen, Tan, Pamela, Wong, Jamie, Wang, Gang, Zimmermann, Tracy, de Fougerolles, Antonin, Vornlocher, Hans-Peter, Langer, Robert, Anderson, Daniel G, Manoharan, Muthiah, Koteliansky, Victor, Horton, Jay D, Fitzgerald, Kevin
Published in Proceedings of the National Academy of Sciences - PNAS (19.08.2008)
Published in Proceedings of the National Academy of Sciences - PNAS (19.08.2008)
Get full text
Journal Article
Oligonucleotide quantification and metabolite profiling by high-resolution and accurate mass spectrometry
Liu, Ju, Li, Jing, Tran, Chris, Aluri, Krishna, Zhang, Xuemei, Clausen, Valerie, Zlatev, Ivan, Guan, Lihua, Chong, Saeho, Charisse, Klaus, Wu, Jing-Tao, Najarian, Diana, Xu, Yuanxin
Published in Bioanalysis (01.11.2019)
Published in Bioanalysis (01.11.2019)
Get full text
Journal Article
5′-(E)-Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA-GalNAc Conjugates
Parmar, Rubina, Willoughby, Jennifer L. S., Liu, Jingxuan, Foster, Donald J., Brigham, Benjamin, Theile, Christopher S., Charisse, Klaus, Akinc, Akin, Guidry, Erin, Pei, Yi, Strapps, Walter, Cancilla, Mark, Stanton, Matthew G., Rajeev, Kallanthottathil G., Sepp-Lorenzino, Laura, Manoharan, Muthiah, Meyers, Rachel, Maier, Martin A., Jadhav, Vasant
Published in Chembiochem : a European journal of chemical biology (02.06.2016)
Published in Chembiochem : a European journal of chemical biology (02.06.2016)
Get full text
Journal Article
4′‑C‑Methoxy-2′-deoxy-2′-fluoro Modified Ribonucleotides Improve Metabolic Stability and Elicit Efficient RNAi-Mediated Gene Silencing
Malek-Adamian, Elise, Guenther, Dale C, Matsuda, Shigeo, Martínez-Montero, Saúl, Zlatev, Ivan, Harp, Joel, Burai Patrascu, Mihai, Foster, Donald J, Fakhoury, Johans, Perkins, Lydia, Moitessier, Nicolas, Manoharan, Rajar M, Taneja, Nate, Bisbe, Anna, Charisse, Klaus, Maier, Martin, Rajeev, Kallanthottathil G, Egli, Martin, Manoharan, Muthiah, Damha, Masad J
Published in Journal of the American Chemical Society (18.10.2017)
Published in Journal of the American Chemical Society (18.10.2017)
Get full text
Journal Article
5′-Morpholino modification of the sense strand of an siRNA makes it a more effective passenger
Kumar, Pawan, Parmar, Rubina G, Brown, Christopher R, Willoughby, Jennifer L. S, Foster, Donald J, Babu, I. Ramesh, Schofield, Sally, Jadhav, Vasant, Charisse, Klaus, Nair, Jayaprakash K, Rajeev, Kallanthottathil G, Maier, Martin A, Egli, Martin, Manoharan, Muthiah
Published in Chemical communications (Cambridge, England) (25.04.2019)
Published in Chemical communications (Cambridge, England) (25.04.2019)
Get full text
Journal Article
Structural basis for the synergy of 4′- and 2′-modifications on siRNA nuclease resistance, thermal stability and RNAi activity
Harp, Joel M, Guenther, Dale C, Bisbe, Anna, Perkins, Lydia, Matsuda, Shigeo, Bommineni, Gopal R, Zlatev, Ivan, Foster, Donald J, Taneja, Nate, Charisse, Klaus, Maier, Martin A, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Egli, Martin
Published in Nucleic acids research (19.09.2018)
Published in Nucleic acids research (19.09.2018)
Get full text
Journal Article
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
Soutschek, Jürgen, Akinc, Akin, Bramlage, Birgit, Charisse, Klaus, Constien, Rainer, Donoghue, Mary, Elbashir, Sayda, Geick, Anke, Hadwiger, Philipp, Harborth, Jens, John, Matthias, Kesavan, Venkitasamy, Lavine, Gary, Pandey, Rajendra K, Racie, Timothy, Rajeev, Kallanthottathil G, Röhl, Ingo, Toudjarska, Ivanka, Wang, Gang, Wuschko, Silvio, Bumcrot, David, Koteliansky, Victor, Limmer, Stefan, Manoharan, Muthiah, Vornlocher, Hans-Peter
Published in Nature (11.11.2004)
Published in Nature (11.11.2004)
Get full text
Journal Article
The RNA-binding protein vigilin regulates VLDL secretion through modulation of Apob mRNA translation
Mobin, Mehrpouya B., Gerstberger, Stefanie, Teupser, Daniel, Campana, Benedetta, Charisse, Klaus, Heim, Markus H., Manoharan, Muthiah, Tuschl, Thomas, Stoffel, Markus
Published in Nature communications (26.09.2016)
Published in Nature communications (26.09.2016)
Get full text
Journal Article
Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification
Chan, Amy, Liebow, Abigail, Yasuda, Makiko, Gan, Lin, Racie, Tim, Maier, Martin, Kuchimanchi, Satya, Foster, Don, Milstein, Stuart, Charisse, Klaus, Sehgal, Alfica, Manoharan, Muthiah, Meyers, Rachel, Fitzgerald, Kevin, Simon, Amy, Desnick, Robert J, Querbes, William
Published in Molecular therapy. Nucleic acids (2015)
Published in Molecular therapy. Nucleic acids (2015)
Get full text
Journal Article
Discovery of a novel deaminated metabolite of a single-stranded oligonucleotide in vivo by mass spectrometry
Li, Jing, Liu, Ju, Enders, Jennifer, Arciprete, Michael, Tran, Chris, Aluri, Krishna, Guan, Li-Hua, O'Shea, Jonathan, Bisbe, Anna, Charissé, Klaus, Zlatev, Ivan, Najarian, Diana, Xu, Yuanxin
Published in Bioanalysis (01.11.2019)
Published in Bioanalysis (01.11.2019)
Get full text
Journal Article